| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAV vector production: Troublesome host innate responses in another setting
|
Wright, J. Fraser
|
|
|
30 |
C |
p. 332
|
artikel
|
| 2 |
Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation
|
Corti, Manuela
|
|
|
30 |
C |
p. 303-314
|
artikel
|
| 3 |
A method for polyclonal antigen-specific T cell-targeted genome editing (TarGET) for adoptive cell transfer applications
|
Palianina, Darya
|
|
|
30 |
C |
p. 147-160
|
artikel
|
| 4 |
Apolipoprotein A-I vascular gene therapy reduces vein-graft atherosclerosis
|
Bi, Lianxiang
|
|
|
30 |
C |
p. 558-572
|
artikel
|
| 5 |
Bringing gene therapy to regenerative medicine
|
Balmayor, Elizabeth R.
|
|
|
30 |
C |
p. 81-82
|
artikel
|
| 6 |
Combined clarification and affinity capture using magnetic resin enables efficient separation of rAAV5 from cell lysate
|
Turco, Federico
|
|
|
30 |
C |
p. 394-402
|
artikel
|
| 7 |
Correlating physicochemical and biological properties to define critical quality attributes of a rAAV vaccine candidate
|
Kumar, Prashant
|
|
|
30 |
C |
p. 103-121
|
artikel
|
| 8 |
CRISPR-Cas9 correction in the DMD mouse model is accompanied by upregulation of Dp71f protein
|
Egorova, Tatiana V.
|
|
|
30 |
C |
p. 161-180
|
artikel
|
| 9 |
CRISPR-Cas9 homology-independent targeted integration of exons 1–19 restores full-length dystrophin in mice
|
Stephenson, Anthony A.
|
|
|
30 |
C |
p. 486-499
|
artikel
|
| 10 |
CRISPR-Cas9-mediated exon skipping as a cardioprotective strategy in Duchenne muscular dystrophy
|
Wilton-Clark, Harry
|
|
|
30 |
C |
p. 500-501
|
artikel
|
| 11 |
Crossing the gates of Babylon: Brain-penetrating enzyme replacement for lysosomal disorders
|
Baldo, Guilherme
|
|
|
30 |
C |
p. 315-316
|
artikel
|
| 12 |
Detection and quantification of integrated vector copy number by multiplex droplet digital PCR in dual-transduced CAR T cells
|
Wang, Wei
|
|
|
30 |
C |
p. 403-410
|
artikel
|
| 13 |
Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6
|
Mellen, Russell W.
|
|
|
30 |
C |
p. 48-64
|
artikel
|
| 14 |
Development of an in vitro genotoxicity assay to detect retroviral vector-induced lymphoid insertional mutants
|
Bastone, Antonella L.
|
|
|
30 |
C |
p. 515-533
|
artikel
|
| 15 |
Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes
|
Kok, Cindy Y.
|
|
|
30 |
C |
p. 459-473
|
artikel
|
| 16 |
Dual-AAV vector-mediated expression of MYO7A improves vestibular function in a mouse model of Usher syndrome 1B
|
Lau, Samantha C.
|
|
|
30 |
C |
p. 534-545
|
artikel
|
| 17 |
Efficacy and muscle safety assessment of fukutin-related protein gene therapy
|
Benasutti, Halli
|
|
|
30 |
C |
p. 65-80
|
artikel
|
| 18 |
Efficient gene transduction in pigs and macaques with the engineered AAV vector AAV.GT5 for hemophilia B gene therapy
|
Kashiwakura, Yuji
|
|
|
30 |
C |
p. 502-514
|
artikel
|
| 19 |
Efficient manufacturing and engraftment of CCR5 gene-edited HSPCs following busulfan conditioning in nonhuman primates
|
Murray, Jason
|
|
|
30 |
C |
p. 276-287
|
artikel
|
| 20 |
Establishment of UGT1A1-knockout human iPS-derived hepatic organoids for UGT1A1-specific kinetics and toxicity evaluation
|
Shintani, Tomohiro
|
|
|
30 |
C |
p. 429-442
|
artikel
|
| 21 |
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy
|
Le Guiner, Caroline
|
|
|
30 |
C |
p. 30-47
|
artikel
|
| 22 |
Expression of gain-of-function CFTR in cystic fibrosis airway cells restores epithelial function better than wild-type or codon-optimized CFTR
|
Woodall, Maximillian
|
|
|
30 |
C |
p. 593-605
|
artikel
|
| 23 |
Fully closed cell sorter for immune cell therapy manufacturing
|
Matsumoto, Masahiro
|
|
|
30 |
C |
p. 367-376
|
artikel
|
| 24 |
Gene replacement therapy in two Golgi-retained CMT1X mutants before and after the onset of demyelinating neuropathy
|
Kagiava, Alexia
|
|
|
30 |
C |
p. 377-393
|
artikel
|
| 25 |
Goody two plasmids: An optimized transient transfection system for AAV vector production
|
Moreno Velasquez, Sergio David
|
|
|
30 |
C |
p. 191-193
|
artikel
|
| 26 |
Human retina-in-a-dish: Unlocking the potential to study mechanisms of inherited retinal disease
|
Yee, Tiffany
|
|
|
30 |
C |
p. 573-575
|
artikel
|
| 27 |
Impact of an autophagy-inducing peptide on immunogenicity and protection efficacy of an adenovirus-vectored SARS-CoV-2 vaccine
|
Sayedahmed, Ekramy E.
|
|
|
30 |
C |
p. 194-207
|
artikel
|
| 28 |
In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans
|
Schwartz, Maura K.
|
|
|
30 |
C |
p. 16-29
|
artikel
|
| 29 |
Inducible HEK293 AAV packaging cell lines expressing Rep proteins
|
Jalšić, Lovro
|
|
|
30 |
C |
p. 259-275
|
artikel
|
| 30 |
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy
|
Emami, Michael R.
|
|
|
30 |
C |
p. 90-102
|
artikel
|
| 31 |
iPSC-derived mesenchymal stem cells attenuate cerebral ischemia-reperfusion injury by inhibiting inflammatory signaling and oxidative stress
|
Arakawa, Masafumi
|
|
|
30 |
C |
p. 333-349
|
artikel
|
| 32 |
Lentiviral delivered aflibercept OXB-203 for treatment of neovascular AMD
|
Iqball, Sharifah
|
|
|
30 |
C |
p. 350-366
|
artikel
|
| 33 |
Lipid nanoparticle mRNA systems containing high levels of sphingomyelin engender higher protein expression in hepatic and extra-hepatic tissues
|
Chander, Nisha
|
|
|
30 |
C |
p. 235-245
|
artikel
|
| 34 |
Mechanistic modeling explains the production dynamics of recombinant adeno-associated virus with the baculovirus expression vector system
|
Destro, Francesco
|
|
|
30 |
C |
p. 122-146
|
artikel
|
| 35 |
Modifying immune responses to adeno-associated virus vectors by capsid engineering
|
Bentler, Martin
|
|
|
30 |
C |
p. 576-592
|
artikel
|
| 36 |
Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease
|
Xu, Liwen
|
|
|
30 |
C |
p. 317-331
|
artikel
|
| 37 |
Necessity of strengthening the current clinical regulatory for companion diagnostics: An institutional comparison of the FDA, EMA, and MFDS
|
Kang, Su Lim
|
|
|
30 |
C |
p. 447-458
|
artikel
|
| 38 |
Novel mutations in UL24 and gH rescue efficient infection of an HSV vector retargeted to TrkA
|
Marzulli, Marco
|
|
|
30 |
C |
p. 208-220
|
artikel
|
| 39 |
Optimizing and developing a scalable, chemically defined, animal component-free lentiviral vector production process in a fixed-bed bioreactor
|
Fiol, Carme Ripoll
|
|
|
30 |
C |
p. 221-234
|
artikel
|
| 40 |
Partial genome content within rAAVs impacts performance in a cell assay-dependent manner
|
Troxell, Bryan
|
|
|
30 |
C |
p. 288-302
|
artikel
|
| 41 |
Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec
|
Asher, Damon
|
|
|
30 |
C |
p. 474-483
|
artikel
|
| 42 |
Prevention of early-onset cardiomyopathy in Dmd exon 52–54 deletion mice by CRISPR-Cas9-mediated exon skipping
|
Rok, Matthew
|
|
|
30 |
C |
p. 246-258
|
artikel
|
| 43 |
Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo
|
Asperti, Claudia
|
|
|
30 |
C |
p. 546-557
|
artikel
|
| 44 |
Selection of viral capsids and promoters affects the efficacy of rescue of Tmprss3-deficient cochlea
|
Aaron, Ksenia A.
|
|
|
30 |
C |
p. 413-428
|
artikel
|
| 45 |
Slow and steady wins the race: Optimizing lentiviral vector production for efficient clinical-scale manufacturing
|
McCarron, Alexandra
|
|
|
30 |
C |
p. 484-485
|
artikel
|
| 46 |
Starburst amacrine cells amplify optogenetic visual restoration through gap junctions
|
Katada, Yusaku
|
|
|
30 |
C |
p. 1-13
|
artikel
|
| 47 |
Swapping the serotype: A novel helper-dependent adenoviral vector platform for in vivo HSC gene therapy
|
Klatt, Denise
|
|
|
30 |
C |
p. 14-15
|
artikel
|
| 48 |
Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia
|
Chandler, Randy J.
|
|
|
30 |
C |
p. 181-190
|
artikel
|
| 49 |
The p53 challenge of hematopoietic stem cell gene editing
|
Dorset, Sofie R.
|
|
|
30 |
C |
p. 83-89
|
artikel
|
| 50 |
Toward effective hematopoietic stem cell gene therapies: Optimized conditioning regimen and stem cell source in harmony
|
Demirci, Selami
|
|
|
30 |
C |
p. 411-412
|
artikel
|
| 51 |
Toward lentiviral vectors for antiangiogenic ocular gene therapy
|
Askou, Anne Louise
|
|
|
30 |
C |
p. 443-446
|
artikel
|
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